University of Ottawa

Purpose

Potent and specific gene therapies used to treat patients have been limited by the lack of delivery vehicles to introduce them into specific cells. Evidence suggests that vesicles naturally produced by cells, called extracellular vesicles, can be highly efficient delivery vehicles. The Gibbings lab has uncovered technology allowing loading of RNA-based gene therapies, such as silencing RNAs, into extracellular vesicles. To optimize the therapeutic use of these extracellular vesicles, this Project will use single-domain or monoclonal antibodies developed by the NRC against novel cell-specific markers to target extracellular vesicles to specific cell types or tissues in vivo, including into immune cells and across the blood brain barrier. This will enhance the precision targeting of extracellular vesicles to deliver RNA-based gene therapies.